The Patient’s Beneficiary: Those Human Risk Takers In Phase III

Medical Patents Broker Inc.
By Kenneth Pearce, President

The medical patents world is flavored with herbs and spices such as "clinical endpoints," "regulatory pathways," "capital burn rates," and "patent morbidity/mortality," sautéed up with a medical expletive all too common in our business.

Except for the actuaries, it is easy to forget those human subjects volunteering to have a new medical invention invade their bodies. The lawyers likely have a book of rules that each volunteer must agree to, or they are not allowed in the study. In the United States, this is known as a “CYA.”

1. The Larger the Number of Phase III Participants, the Greater the Potential for Those Rare Yet Potentially Deadly Side Effects

Armed with Phase I and II data where only a few people were tested, developers have a reasonable belief about the medical invention’s safety profile. But as we all know, what works well for a few does not always scale well for a multitude.

One of the primary purposes of Phase III is to determine if the outliers are really outliers, or if they are common to a greater percentage of subjects. Some of the results can generate a better life for a patient, while others can create a lifelong physical burden that would have never occurred without the medical invention.

For the unfortunate few subjects who will live the remainder of their lives handicapped by the study, they will never get a do-over. Similar to our wounded veterans, we thank them for their service and move on to the next project.

2. The Coin Toss (Placebos and Active Comparators) For Treating Patients' Current Medical Conditions

In pharmacy school, randomized, "double-blind" Phase III trials—where neither the patient nor the doctor knows who is getting what—were drilled into my head.

When a Phase III trial is approved to treat current patients with a specific disease, the two major risks are:

• The Placebo: With a progressive, painful disease, the subjects risk receiving no active treatment. Yet, some still get better.
• The Active Comparator: If an older drug like 5-fluorouracil is used as the comparator, the side effects alone can almost kill the subject.

3. The Probability of Success

The success rate of Phase III trials is roughly 50% to 60%. These subjects risk increased pain and suffering, lifelong disabilities, and even death for the greater good of the population. Still, the media does not report that they are heroes.

🎽 The President’s Viewpoint

Money and the fear of death are great medical motivators. During my stint as a hospital pharmacist, I was an eyewitness to this phenomenon.

It seems to me like these volunteers should receive far more respect than they do.

It is written that to give one’s life for another is the greatest gift of all.